-
1.发明公开
公开(公告)号：EP3926046A3
公开(公告)日：2022-03-23
申请号：EP21166750.6
申请日：2003-02-20
发明人： MCSWIGGEN, James , BEIGELMAN, Leonid , CHOWRIRA, Bharat , PAVCO, Pamela , FOSNAUGH, Kathy , JAMISON, Sharon , USMAN, Nassim , THOMPSON, James
IPC分类号： C12N15/11 , C12N15/85 , C12N15/86 , C12Q1/68 , A61K48/00 , C12P19/34 , C07H21/02 , C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), doublestranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
-
2.发明公开
公开(公告)号：EP3926046A2
公开(公告)日：2021-12-22
申请号：EP21166750.6
申请日：2003-02-20
发明人： MCSWIGGEN, James , BEIGELMAN, Leonid , CHOWRIRA, Bharat , PAVCO, Pamela , FOSNAUGH, Kathy , JAMISON, Sharon , USMAN, Nassim , THOMPSON, James
IPC分类号： C12N15/11 , C12N15/85 , C12N15/86 , C12Q1/68 , A61K48/00 , C12P19/34 , C07H21/02 , C07H21/04
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), doublestranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
-
公开(公告)号：EP3434667B1
公开(公告)日：2020-11-04
申请号：EP18192871.4
申请日：2013-04-16
IPC分类号： C07C229/12 , C07C229/28 , C07C229/30 , C12N15/113 , A61K9/51
-
公开(公告)号：EP2555777B1
公开(公告)日：2019-02-20
申请号：EP11766550.5
申请日：2011-04-04
发明人： AARONSON, Jeffrey, G. , BARNETT, Stanley, F. , BARTZ, Rene , COLLETTI, Steven, L. , JADHAV, Vasant, R. , MOMOSE, Aaron, A. , SHAW, Anthony, W. , TELLERS, David, M. , TUCKER, Thomas, J. , WANG, Weimin , YUAN, Yu
IPC分类号： A61K47/64 , A61K47/60 , A61K47/55 , A61K31/713 , C07K19/00
-
公开(公告)号：EP3358013A1
公开(公告)日：2018-08-08
申请号：EP17002009.3
申请日：2013-04-26
发明人： Carr, Brian Allen , Jadhav, Vasant R. , Kenski, Denise M. , Tellers, David M. , Willingham, Aarron T.
IPC分类号： C12N15/113 , C12N15/11
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
-
公开(公告)号：EP2830594B1
公开(公告)日：2018-05-09
申请号：EP13767382.8
申请日：2013-03-25
IPC分类号： A61K9/127 , C07C217/28 , A61K47/28 , C12N15/113 , A61K48/00
CPC分类号： C07C217/40 , A61K9/1272 , A61K9/145 , A61K9/146 , A61K9/5123 , A61K9/5146 , A61K31/713 , A61K47/10 , A61K47/14 , A61K47/183 , A61K47/24 , A61K47/28 , C07C217/28 , C07C2601/02 , C12N15/113 , C12N15/88 , C12N2320/32
摘要： Disclosed herein are novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. The cationic lipids can demonstrate enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
-
7.发明授权NOVEL LOW MOLECULAR WEIGHT CYCLIC AMINE CONTAINING CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY 有权用于寡核苷酸递送的含有阳离子脂质的新型低分子量环胺
公开(公告)号：EP2635265B1
公开(公告)日：2018-04-04
申请号：EP11838596.2
申请日：2011-10-31
IPC分类号： C07D207/12 , A61K9/50 , A61K9/127 , A61K31/41 , A61K9/51
CPC分类号： C12N15/1137 , A61K9/5123 , C07D207/12 , C07D211/40 , C07D223/08 , C12N15/113 , C12N2310/14 , C12N2320/32
摘要： The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids comprising at least one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
-
公开(公告)号：EP2609198B8
公开(公告)日：2018-03-28
申请号：EP11820420.5
申请日：2011-08-19
发明人： LIM, Lee , WILLINGHAM, Aarron , ZHAO, Lihong , CHORN, Guillaume
IPC分类号： C12N15/11 , C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1138 , C12N2310/14 , C12N2310/141 , C12N2310/318 , C12N2310/3183 , C12N2310/332 , C12N2310/344 , C12N2310/351 , C12N2310/3515 , C12N2310/3519 , C12N2310/322 , C12N2310/3533 , C12N2310/321 , C12N2310/3521
摘要： Single-stranded RNA molecules comprise one or more internal, non-nucleotide spacers, covalently linked with nucleotide portions of the molecule are provided. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism, and thus represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.
-
9.发明授权RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 有权RNA介导的干扰介导的短基因干扰抑制基因表达（SINA）
公开(公告)号：EP2902406B1
公开(公告)日：2018-01-31
申请号：EP14195627.6
申请日：2003-02-20
发明人： MCSWIGGEN, James , BEIGELMAN, Leonid , CHOWRIRA, Bharat , PAVCO, Pamela , FOSNAUGH, Kathy , JAMISON, Sharon , USMAN, Nassim , THOMPSON, James
CPC分类号： C12N15/8218 , A61K38/00 , A61K47/54 , C07H21/02 , C12N15/111 , C12N15/1131 , C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/344 , C12N2310/346 , C12N2310/53 , C12N2320/51 , C12N2330/30 , Y02A50/393 , C12N2310/3521
摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
-
">10.发明授权POST-SYNTHETIC CHEMICAL MODIFICATION OF RNA AT THE 2'-POSITION OF THE RIBOSE RING VIA "CLICK" CHEMISTRY 有权在2'核糖位置RNA的合成POST化学修饰使用点击化学
公开(公告)号：EP2526113B1
公开(公告)日：2016-08-10
申请号：EP11735064.5
申请日：2011-01-19
发明人： ZEWGE, Daniel , GOSSELIN, Francis
IPC分类号： C07H21/02
CPC分类号： C12N15/113 , C07C217/40 , C07C217/52 , C07C2601/02 , C07D207/08 , C07D207/12 , C07D209/48 , C07D211/22 , C07D211/44 , C07D211/46 , C07D295/088 , C07H21/02 , C12N15/1137 , C12N2310/14 , C12N2310/141 , C12N2310/321 , C12N2330/30
-
-
-
-
-
-
-
-
-
搜索结果
198 条记录 (耗时 0.037 秒)
